I'm Aware That I'm Rare: the phaware® podcast

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Dr. Zamanian is the Director of the Adult Pulmonary
Hypertension (PH) Program at Stanford University Medical Center. In
Part 1 of his 2 episode series, Dr. Zamanian discusses breakthrough
interventions in clinical trials. He currently directs the Vera
Moulton Wall Center clinical database and biobank and focuses his
research on clinical characterization and impact of novel risk
factors such as methamphetamine use, and biomarkers, such as
insulin resistance, in pulmonary arterial hypertension.
Beyond industry clinical trials and registries, Dr. Zamanian
has re-focused the research mission of the Stanford PH program by
collaborating with basic science faculty and implementing several
proof-of-concept and phase II clinical trials of novel therapeutics
developed at Stanford University. Dr. Zamanian also serves on
phaware’s Medical Advisory Board.

I'm Roham Zamanian. I'm the medical director of the pulmonary
vascular disease program at Stanford University Medical Center.

Today we'll talk about clinical trials.

Clinical trials are basically the experiments that prove or
disprove the effectiveness, the safety, the tolerability and really
the utility of either the medications, it could be a procedure, it
could be really anything. A clinical trial is really the accepted
method to testing medical interventions for diseases and conditions
that we deal with in medicine.

So, from inception to completion, it depends on the size and the
scope of a clinical trial, but it's much, much longer than the
conduct of the clinical trial. So for example, for medical clinical
trials, that are testing a therapy in patients, there's often 10 or
15 years of basic and translational research that's already been
done to develop the background. There's often several years of work
to make sure that the intervention that's being tested on humans
will be safe. And then, depends on the scope of the study, and
depends on which disease.

For example, in pulmonary hypertension, the studies that began
at the very beginning of the therapeutic paradigms in pulmonary
hypertension were 12-week studies. It doesn't mean that the whole
study was done in 12 weeks. But those studies tested an
intervention, a medical therapy over 12 weeks time. They're often
preceded by several years of work to develop this clinical trial,
to get the approval, to get the funding for the clinical trial, and
really maybe a year or two after the completion of the clinical
trial, to do the appropriate analysis and submit for
publications.

So those were short days. These days, in pulmonary hypertension,
or even in other diseases, people may be looking over several years
period of time, to look at the effect of their intervention. So
really it just really depends on what disease stage that the
clinical trial is conducted, what the aims are, and what is
acceptable to the community as a whole.

Historically, there's a big body of literature in terms of
ethics of clinical trial conduct. Out of several historical events
in human history, have come sets of understanding and Bill of
Rights for patients who undergo clinical trials.

So to be specific, I'm not sure if you're aware of the Tuskegee
Experiments, where African American men in the Army were infected
with syphilis and were not told about that. And the experiment was
to see the impact of neurosyphilis on human function over a long
period of time without therapy. Now, this came out, it was a big
deal, it was totally unethical. So from these incidents, there is
not a Declaration of Helsinki that provides the framework of
safeguarding patients when they go into clinical trials.

So first of all, there is a major history to that. Second, most
important, is that patients do need to know that for any
appropriate clinical trial to be conducted, in First World
countries, there has to be approval from an institutional review
board, which will review the safety and the appropriateness of the
conduct for that clinical trial.

There is usually, the established method of a data safety
monitoring board. The data safety monitoring board is specific to
the study, that will continue to follow the study as it unrolls,
and make sure that the intervention is appropriate for
patients.

And also, each individual patient is consented, is given a Bill
of Rights, and is given the option at any time to withdraw from the
clinical study. So first of all, there is tremendous and very
important and necessary human protection that's in there for any
clinical trial, whether it's in cardiac, respiratory, other
diseases, cancer, et cetera.

The second part of it is, patients need to recognize, we as a
group of physicians, need to recognize that the way that we can
develop breakthrough interventions for patients is by having
patients involved into the clinical trial. So we take care of the
safety and appropriateness of the clinical trial.

And this might be a little bit controversial, but I tell my
patients that if there is anything that I feel that they might have
a responsibility, it may be the responsibility to be open to
enrolling into clinical trials because I'm sure there are patients
today that are benefiting from participation of patients from 10
years ago. And that their therapies are based on the experiments
that were done on patients from 10 years ago.

So there is a risk benefit. We take huge lengths to protect
subjects against harm. We don't do anything that is inappropriate.
And we must not. At the same time, yes, there is a risk
benefit.

And often my patients ask me, "How about if I go into this study
and I get the placebo?" And that's a conversation we always need to
have with the patients. It's a necessary part of experimentation to
have a control and those controls may be placebos. And for us to
have evidence of either safety or efficacy or whatever is of the
intervention that we're doing.

Yeah, it may be that those patients will not get the supposed
therapeutic benefit of an agent that's being tested. But at the
same time, it's hard to know. If you're testing a drug that hasn't
been proven to be effective, you really never know if the drug is
going to help or not at the end.

So you know, it's a very complicated conversation but I think
what I would want my patients to walk away from the conversations
that I have with them is: A. Your safety is number one priority.
There are many, many institutional and investigator-specific
actions that are taken primarily for the safety of the patients who
are in the clinical trial. The second part of it is, we have do
these clinical trials to be able to offer new potential therapies
or other things that we're testing to our patients. The final point
is, I think we're all in this together.

About the Podcast

A new podcast series devoted to raising global pulmonary hypertension awareness with dynamic stories from PH patients, caregivers and medical professionals from around the world. New Episodes every Monday & Thursday.
Through this series of impactful, insightful and, most importantly, hopeful stories from members of the global pulmonary hypertension community, we hope to further the global #phaware conversation as well as to capture, engage and enable misdiagnosed and undiagnosed PH patients because early diagnosis and treatment can mean the difference between life and death.
Topics range from the importance of early diagnosis and global PH awareness, to the impact of clinical trials to how patient support and advocacy are paramount to battling this disease.
Learn more about PH at www.phaware.global